Shares of Verve Therapeutics (VERV) surged 26.4% on Monday and continue to soar today after the company announced positive initial data from an early-stage study of its investigational candidate, VERVE-102, which is being developed for reducing cholesterol levels. VERVE-102 is a novel, in vivo, investigational base editing medicine.The patient population enrolled in the phase Ib Heart-2 study comprises individuals with heterozygous familial hypercholesterolemia and/or premature coronary artery disease — two ...

SAN DIEGO, April 14, 2025 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ:FATE), a clinical- stage biopharmaceutical company dedicated to bringing a pipeline of induced pluripotent stem cell (iPSC)- derived off-the-shelf cellular immunotherapies to patients, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to FT819, an investigational, off-the-shelf, iPSC-derived CAR T-cell therapy in Phase 1 clinical development for the t ...

Core Insights - Verve Therapeutics announced positive initial data from the Heart-2 Phase 1b clinical trial of VERVE-102, showing significant reductions in LDL-C and PCSK9 levels with a single infusion [1][2][10] Efficacy - A single infusion of VERVE-102 resulted in a mean LDL-C reduction of 53% and a maximum reduction of 69% in the 0.6 mg/kg dose cohort [1][11] - The trial demonstrated a dose-dependent response, with mean LDL-C reductions of 21% at 0.3 mg/kg, 41% at 0.45 mg/kg, and 53% at 0.6 mg/kg [11] - Among participants receiving a total RNA dose greater than 50 mg, a mean LDL-C reduction of 59% was observed [9][10] Safety and Tolerability - VERVE-102 was well-tolerated with no treatment-related serious adverse events and no clinically significant changes in liver enzymes or platelets [1][6] - One infusion-related reaction was reported, which was transient and resolved with acetaminophen [6] Clinical Trial Design - The Heart-2 trial is an open-label Phase 1b study evaluating the safety and tolerability of VERVE-102 in patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD) [4][5] - The trial includes four dose cohorts, with initial data reported from 14 participants across the first three cohorts [5] Future Plans - Verve plans to report final Heart-2 dose escalation data and initiate a Phase 2 clinical trial for VERVE-102 in the second half of 2025 [1][13] - The company expects to receive a decision from Eli Lilly regarding the PCSK9 opt-in in the second half of 2025 [14] Company Overview - Verve Therapeutics is focused on developing genetic medicines for cardiovascular disease, with VERVE-102 targeting the PCSK9 gene to reduce LDL-C levels [16] - The company aims to transform treatment from chronic therapies to single-course gene editing medicines [16]

Core Insights - IDEAYA Biosciences announced a successful FDA Type D meeting regarding the Phase 3 trial design for darovasertib as a neoadjuvant therapy for primary uveal melanoma (UM) [1][2] - The trial aims to assess safety and efficacy with primary endpoints focused on eye preservation and vision loss, alongside secondary endpoints related to Event-Free Survival (EFS) [2][6] Company Overview - IDEAYA Biosciences is a precision medicine oncology company dedicated to developing targeted therapeutics through molecular diagnostics [8] - The company integrates capabilities in identifying translational biomarkers with drug discovery to optimize patient selection for its therapies [8] Drug Development Details - Darovasertib is a selective protein kinase C (PKC) inhibitor targeting both primary and metastatic UM [3] - The drug has received Breakthrough Therapy Designation and Fast Track designation from the FDA for its use in neoadjuvant therapy and in combination with crizotinib for metastatic UM [3] Phase 3 Trial Design - The Phase 3 trial is projected to enroll approximately 520 patients, randomized in a 2:1 ratio between darovasertib and control [5][6] - Two cohorts will be included: 120 enucleation-eligible UM patients and 400 plaque brachytherapy (PB) eligible UM patients [5][6] Clinical Endpoints - Primary endpoints include eye preservation rate for enucleation patients and vision loss for PB patients, with no detriment to EFS as a secondary endpoint [6][7] - Additional secondary endpoints will assess overall response rate, macular edema, and vision loss metrics [7] Regulatory Pathway - The company plans to initiate the Phase 3 trial in the first half of 2025, with potential for earlier regulatory submission of enucleation cohort data [4][11]

Core Insights - Verve Therapeutics has received Fast Track designation from the FDA for its investigational drug VERVE-102, aimed at treating hyperlipidemia and reducing LDL-C levels in patients at high cardiovascular risk [1][2] - VERVE-102 is designed as a single-course treatment that permanently disables the PCSK9 gene in the liver, potentially transforming the management of atherosclerotic cardiovascular disease [1][5] - The company is currently conducting the Phase 1b Heart-2 clinical trial to assess the safety and tolerability of VERVE-102 in patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease [1][3] Company Overview - Verve Therapeutics is a clinical-stage company focused on developing genetic medicines for cardiovascular diseases, with a goal to shift treatment from chronic therapies to single-course gene editing solutions [5] - The company's lead programs include VERVE-102, VERVE-201, and VERVE-301, targeting key cholesterol drivers associated with atherosclerosis [5] - VERVE-201 aims to turn off the ANGPTL3 gene for patients with refractory hypercholesterolemia, while VERVE-301 targets the LPA gene to reduce Lp(a) levels, an independent risk factor for cardiovascular diseases [5] Clinical Trial Updates - Initial demographic and safety data from the Heart-2 clinical trial are expected to be announced in Q2 2025, including results from three dose cohorts [3] - The final data for the dose escalation portion of the Heart-2 trial is anticipated in the second half of 2025, along with the initiation of the Phase 2 clinical trial for the PCSK9 program [4]

Core Viewpoint - The merger between Renovaro Biosciences Inc. and BioSymetrics aims to enhance advanced AI-driven drug development and expand precision medicine applications in oncology and neurology [1][4]. Group 1: Merger Details - Renovaro completed its merger with BioSymetrics on April 8, 2025, to strengthen its capabilities in data repository and biomarker discovery [2][1]. - The merger integrates BioSymetrics' Elion platform, which utilizes AI and machine learning to accelerate the discovery of diagnostics and therapeutics [3][4]. Group 2: Technological Synergy - BioSymetrics' Phenograph and advanced in vivo modeling systems are designed to improve target and biomarker identification, enabling patient stratification and drug repurposing [3][4]. - The combined company aims to streamline the translation of biomarker insights into accelerated drug discovery timelines, enhancing research efficiency [3][5]. Group 3: Strategic Goals - The merger is expected to enhance Renovaro's ability to identify new therapeutic targets and validate diagnostics, particularly in oncology and neurology [4][5]. - The focus will be on integrating AI-powered biomarker discovery with innovative drug development to improve patient outcomes globally [5].

HOUSTON, April 02, 2025 (GLOBE NEWSWIRE) -- FibroBiologics, Inc. (NASDAQ:FBLG) ("FibroBiologics"), a clinical-stage biotechnology company with 240+ patents issued and pending for the development of therapeutics and potential cures for chronic diseases using fibroblasts and fibroblast-derived materials, today announced a significant advancement in neurodegenerative disease research. Leveraging the well-established Cuprizone animal model, FibroBiologics has demonstrated that intravenous fibroblasts can facili ...

- Veteran Industry Executive Brings Decades of Financial and Leadership Experience - ANN ARBOR, Mich., April 01, 2025 (GLOBE NEWSWIRE) -- Esperion (NASDAQ: ESPR) today announced the appointment of Robert E. Hoffman as an independent director, effective immediately, and he will also serve as Chairperson of the Audit Committee effective May 29, 2025. In addition, the Company announced that Nicole Vitullo and Antonio M. Gotto, Jr., M.D., D. Phil. will step down from the board of directors, effective June 1, 20 ...

Core Insights - Korro Bio, Inc. has appointed Dr. Loïc Vincent as Chief Scientific Officer, bringing over 20 years of drug development experience to the company [2][3] - The company is advancing multiple candidates in its pipeline, including KRRO-110, currently in Phase 1/2a clinical study for Alpha-1 Antitrypsin Deficiency [2] - Korro's RNA-editing platform aims to develop genetic medicines for both rare and prevalent diseases, enhancing precision and long-term tolerability [4] Company Overview - Korro Bio is a clinical-stage biopharmaceutical company focused on RNA editing to create genetic medicines [4] - The company utilizes an oligonucleotide-based approach to expand the reach of genetic therapies, leveraging established regulatory pathways [4] - Korro is headquartered in Cambridge, Massachusetts [4] Leadership Appointments - Dr. GaoZhong Zhu has joined as Senior Vice President of Chemistry, Manufacturing and Controls, with over 25 years of experience in pharmaceutical development [5] - Oliver Dolan has been promoted to Principal Accounting Officer, having served as Senior Vice President of Finance since January 2024 [5]

Humacyte (HUMA) Q4 2024 Earnings Call March 28, 2025 04:23 PM ET Company Participants Tom Johnson - Director - Corporate CommunicationsLaura Niklason - Founder, President, CEO & DirectorDale Sander - CFO, Chief Corporate Development Officer & TreasurerJoshua Jennings - Managing DirectorVernon Bernardino - Managing Director Conference Call Participants Ryan Zimmerman - Managing Director & Medical Technology AnalystNone - AnalystBruce Jackson - Senior Analyst Operator morning, ladies and gentlemen, and welcom ...